Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial von Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

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Ketone Bodies Mediate Antiseizure Effects von Krueger, Jena M, Nordli, Jr, Douglas R

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Remission of Childhood-onset Epilepsy von Krueger, Jena M, Nordli, Jr, Douglas R

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Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study von Crawford, Thomas O, Day, John W, De Vivo, Darryl C, Krueger, Jena M, Mercuri, Eugenio, Nascimento, Andres, Pasternak, Amy, Mazzone, Elena Stacy, Duong, Tina, Song, Guochen, Marantz, Jing L, Baver, Scott, Yu, Dongzi, Liu, Lan, Darras, Basil T

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Incidence of Dravet Syndrome in a US Population von Krueger, Jena, Berg, Anne T

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Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care von Henderson, Marian L, Zieba, Jacob K, Li, Xiaopeng, Campbell, Daniel B, Williams, Michael R, Vogt, Daniel L, Bupp, Caleb P, Edgerly, Yvonne M, Rajasekaran, Surender, Hartog, Nicholas L, Prokop, Jeremy W, Krueger, Jena M

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A treatable metabolic cause of encephalopathy: cobalamin C deficiency in an 8-year-old male von Krueger, Jena M, Piantino, Juan, Smith, Craig M, Angle, Brad, Venkatesan, Charu, Wainwright, Mark S

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Epilepsy Following Neonatal Seizures Secondary to Hemorrhagic Stroke in Term Neonates von Venkatesan, Charu, Millichap, John J., Krueger, Jena M., Nangia, Srishti, Ritacco, David G., Stack, Cynthia, Nordli, Douglas R.

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Prognosis in Pediatric Myasthenia Gravis von Krueger, Jena

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Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2 (S39.004) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Finkel, Richard, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, McGill, Bryan, Macek, Thomas

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Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with Three Copies of SMN2 (P15-5.001) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Finkel, Richard, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, McGill, Bryan, Macek, Thomas

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2 (4163) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Thomas

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Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 2 Copies of SMN2 (4190) von Strauss, Kevin, Muntoni, Francesco, Farrar, Michelle, Saito, Kayoko, Mendell, Jerry, Servais, Laurent, McMillan, Hugh, Swoboda, Kathryn, Kwon, Jennifer, Zaidman, Craig, Chiriboga, Claudia, Iannaccone, Susan, Krueger, Jena, Parsons, Julie, Shieh, Perry, Kavanagh, Sarah, Chand, Deepa, Tauscher-Wisniewski, Sitra, Macek, Tom

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Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations von Schroth, Mary K, Deans, Jennifer, Bharucha Goebel, Diana X, Burnette, W Bryan, Darras, Basil T, Elsheikh, Bakri H, Felker, Marcia V, Klein, Andrea, Krueger, Jena, Proud, Crystal M, Veerapandiyan, Aravindhan, Graham, Robert J

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Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update (2384) von Strauss, Kevin A., Farrar, Michelle A., Swoboda, Kathryn J., Saito, Kayoko, Chiriboga, Claudia A., Finkel, Richard S., Iannaccone, Susan T., Krueger, Jena M., Kwon, Jennifer M., McMillan, Hugh J., Servais, Laurent, Mendell, Jerry R., Parsons, Julie, Scoto, Mariacristina, Shieh, Perry B., Zaidman, Craig, Schultz, Meredith, Kernbauer, Elaine, Joshi, Shivani, Ogrinc, Francis G., Kavanagh, Sarah, Feltner, Douglas E., Tauscher-Wisniewski, Sitra, McGill, Bryan E., Sproule, Douglas M., Muntoni, Francesco

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Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians von Veerapandiyan, Aravindhan, Connolly, Anne M., Mathews, Katherine D., Nelson, Stanley, McDonald, Craig, Finkel, Richard S., Vedanarayanan, Vettaikorumakankav, Tian, Cuixia, Apkon, Susan, Parsons, Julie A., Soslow, Jonathan H., Burnette, William Bryan, Batley, Kaitlin Y., Iannaccone, Susan T., Rocha, Carolina Tesi, Flanigan, Kevin M., Bharucha‐Goebel, Diana, Wright, Sarah, Monduy, Migvis, Treidler, Simona, Kumar, Ashutosh, Kuntz, Nancy L., Rao, Vamshi K., Schrader, Rachel, Bernes, Saunder M., Stefans, Vikki Ann, Krueger, Jena M., Felker, Marcia V., Hamid, Omer Abdul, Lakhotia, Arpita, Matesanz, Susan, Ghosh, Partha S., Katz, Natalie, Abdel‐Hamid, Hoda, Laverty, Chamindra G., Lee, Bo Hoon, Harper, Amy, Ramos‐Platt, Leigh, Castro, Diana, Butterfield, Russell J., Proud, Crystal M., Zaidman, Craig M., Ciafaloni, Emma

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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial von Strauss, Kevin A, Farrar, Michelle A, Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R, Servais, Laurent, McMillan, Hugh J, Finkel, Richard S, Swoboda, Kathryn J, Kwon, Jennifer M, Zaidman, Craig M, Chiriboga, Claudia A, Iannaccone, Susan T, Krueger, Jena M, Parsons, Julie A, Shieh, Perry B, Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E, Macek, Thomas A

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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial von Strauss, Kevin A, Farrar, Michelle A, Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R, Servais, Laurent, McMillan, Hugh J, Finkel, Richard S, Swoboda, Kathryn J, Kwon, Jennifer M, Zaidman, Craig M, Chiriboga, Claudia A, Iannaccone, Susan T, Krueger, Jena M, Parsons, Julie A, Shieh, Perry B, Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E, Macek, Thomas A

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Motorrad-Sandbahnrennen in Panitzsch. Motorradfahrer Lothar Werner und ?,Krüger (Jena) im Beiwagengespann von Rössing, Roger & Rössing, Renate (Herstellung) (Fotograf)

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